Base editing of the HBG promoter induces potent fetal hemoglobin expression with no detectable off-target mutations in human HSCs-Reference-Cited by-全球学者库

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Base editing of the HBG promoter induces potent fetal hemoglobin expression with no detectable off-target mutations in human HSCs

Published:2023-12 Issue:12 Volume:30 Page:1624-1639.e8
ISSN:1934-5909
Container-title:Cell Stem Cell
language:en
Short-container-title:Cell Stem Cell

Author:

Han Wenyan,Qiu Hou-Yuan,Sun Shangwu,Fu Zhi-Can,Wang Guo-Quan,Qian Xiaowen,Wang Lijie,Zhai Xiaowen,Wei Jia,Wang Yichuan,Guo Yi-Lin,Cao Guo-Hua,Ji Rui-Jin,Zhang Yi-Zhou,Ma Hongxia,Wang Hongsheng,Zhao Mingli,Wu Jing,Bi Lili,Chen Qiu-Bing,Li Zifeng,Yu Ling,Mou Xiaodun,Yin Hao,Yang Li,Chen Jia,Yang Bei,Zhang Ying^ORCID

Publisher

Elsevier BV

Subject

Cell Biology,Genetics,Molecular Medicine

Reference83 articles.

1. Current advances of CRISPR-Cas technology in cell therapy;Qiu;Cell Insight,2022

2. Gene therapy using haematopoietic stem and progenitor cells;Ferrari;Nat. Rev. Genet.,2021

3. Ready for repair? Gene editing enters the clinic for the treatment of human disease;Ernst;Mol. Ther. Methods Clin. Dev.,2020

4. Gene replacement of alpha-globin with beta-globin restores hemoglobin balance in beta-thalassemia-derived hematopoietic stem and progenitor cells;Cromer;Nat. Med.,2021

5. Transcriptional regulation of fetal to adult hemoglobin switching: new therapeutic opportunities;Wilber;Blood,2011

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